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- **RGX-202 Milestone**: Pivotal trial enrollment complete; BLA submission in mid-2026 with potential accelerated approval. Confirmatory trial enrolling 30 patients to support full approval.
- **MPS II Progress**: FDA inspections cleared; BLA on track for February 2026 PDUFA date. Commercial doses via Nippon Shinyaku expected early 2026.
- **Financial Strength**: $302 million cash balance with runway into 2027, supported by $145 million healthcare royalty monetization and $110 million Nippon Shinyaku payment.
- **Retinal Leadership**: Wet AMD trial largest in gene therapy history; DR program to initiate Phase IIb/III in 2026 with AbbVie collaboration.

**【CEO Summary Section】**
- **RGX-202 Duchenne Program**: Completed enrollment in the pivotal AFFINITY trial, with plans to submit a BLA under accelerated approval in mid-2026. The program features a novel immune suppression regimen, high-purity manufacturing (80% full capsids), and a 2,500 doses/year commercial capacity. Confirmatory trial enrollment is progressing, targeting 30 patients.
- **RGX-121 MPS II Program**: FDA inspections of clinical sites and manufacturing facilities resulted in no observations. The BLA is on track for a February 8, 2026, PDUFA date, with potential commercial doses available early 2026 via partner Nippon Shinyaku.
- **Retinal Franchise**: Wet AMD programs (sura-vec) achieved full enrollment in the largest gene therapy trial (1,200+ patients). Diabetic retinopathy (DR) program expects to initiate Phase IIb/III trials in early 2026, leveraging AbbVie's global infrastructure.

**【CFO Financial Commentary】**
- **Cash Position**: $302 million as of Q3 2025, up from $245 million in Q4 2024, driven by $110 million from Nippon Shinyaku and $145 million from healthcare royalty monetization. Cash runway extends into early 2027, with potential extensions via priority review voucher sales, milestones, and partnerships.
- **Revenue Growth**: Q3 2025 revenue of $30 million (vs. $24 million in Q3 2024) reflects increasing collaboration activity, particularly with AbbVie and Nippon Shinyaku.

**【Q&A Key Highlights】**
- **Regulatory Strategy**: RGX-202 eligible for accelerated approval based on functional benefits exceeding natural history controls. FDA interactions for MPS II were productive, with no additional data requests.
- **Manufacturing Scale**: Rockville facility equipped with a 2,000-liter bioreactor, capable of producing 2,500 RGX-202 doses/year. MPS II uses <5% of total capacity, enabling focus on high-priority programs.
- **Commercial Partnerships**: AbbVie's global ophthalmology expertise accelerates retinal programs, with $2 of every $3 in wet AMD costs covered by AbbVie. Nippon Shinyaku's MPS II partnership includes a $100 million milestone for DR first patient dosing.
- **Risk Considerations**: Duchenne label may require non-ambulatory patient studies post-launch. Medicare coverage for gene therapies is evolving, with AbbVie leading wet AMD reimbursement discussions.

REGENXBIO Inc. (RGNX) Q3 2025 Earnings Call Transcript

- **RGX-202 Lead**: Duchenne gene therapy enrollment accelerates to October 2025, with 80%+ capsid purity and no safety issues in Phase I/II. Commercial manufacturing begins Q4 2025.
- **Sura-vec Expansion**: Diabetic retinopathy Phase IIb/III trial initiated with $100M milestone. 50% of patients achieved 2+ DRSS improvement without injections in 2 years.
- **RGX-121 Milestone**: Hunter syndrome BLA accepted with November 2025 PDUFA date, positioning for 2026 launch.
- **Financial Strength**: $364M cash runway to early 2027, supported by $100M AbbVie milestone and non-dilutive financing options.

**【CEO Summary Section】**
- **RGX-202 Duchenne Update**: Accelerated enrollment for pivotal trial to complete by October 2025, with 30 patients. Commercial manufacturing begins Q4 2025 at Rockville facility (2,500 doses/year). Phase I/II data shows functional improvement in 8+ year olds with no safety issues. Proactive immune suppression regimen and 80%+ full capsid purity differentiate from competitors.
- **Sura-vec (ABBV-RGX-314) Expansion**: Phase IIb/III trial for diabetic retinopathy (DR) initiated with $100M milestone payment. 2-year data shows 50% of dose level 3 patients achieved 2+ DRSS improvement without supplemental injections. Wet AMD subretinal trials nearing completion with topline data expected 2026.
- **RGX-121 Hunter Syndrome**: BLA accepted under accelerated pathway with PDUFA date November 9, 2025. First commercial product on track for early 2026 launch with Nippon Shinyaku.

**【CFO Financial Commentary】**
- **Q2 2025 Financials**: Cash balance $364M (up from $245M YoY) driven by $110M Nippon Shinyaku upfront and $145M royalty monetization. R&D expenses $60M (vs $49M YoY) due to manufacturing and clinical costs.
- **Sura-vec Collaboration**: $100M milestone payment for Phase IIb/III initiation. AbbVie covers $200M pivotal program cost through two $100M milestones. Current cash runway extends to early 2027 with multiple non-dilutive financing options.

**【Q&A Highlights】**
- **Duchenne Community Sentiment**: High enrollment interest despite recent safety concerns, driven by short-course immune suppression regimen transparency. 80%+ full capsid purity reduces vector load and safety risks.
- **DR Trial Design**: Phase IIb/III combines dose selection (levels 3/4) with 1-year primary endpoint. 50% efficacy without injections vs 6x historical control. 2-year durability data supports single administration benefit.
- **FDA Interactions**: Positive engagement on RGX-121 with no observations in pre-BLA inspections. Duchenne program benefits from urgent unmet need despite CBER leadership changes.

REGENXBIO Inc. (RGNX) Q2 2025 Earnings Call Transcript

- **RGX-121 Regulatory Momentum**: BLA acceptance imminent; potential FDA approval in H2 2025 under accelerated pathway.
- **RGX-202 Clinical Velocity**: 50% enrollment in pivotal Duchenne trial; commercial supply production starts Q3 2025.
- **RGX-314 Retina Dominance**: First-in-class gene therapy for wet AMD; 4-year durability data; DR Phase III on track for 2025 start.
- **Financial Strength**: $272M cash; non-dilutive financing options extend runway beyond 2026.
- **Manufacturing Excellence**: Industry-leading 80%+ full capsid purity in RGX-202; scalable 2,500 doses/year capacity.


**【CEO Summary Section】**
- RGX-121 BLA submission under accelerated approval pathway; FDA acceptance expected imminently, with potential approval in H2 2025.
- RGX-202 Duchenne trial enrollment surpasses 50%, targeting 30 patients by end-2025; commercial supply production to begin Q3 2025.
- RGX-314 wet AMD pivotal trials (subretinal) on track for enrollment completion in 2025, with DR Phase III preparation underway.

**【CFO Financial Update】**
- Q1 2025 cash balance: $272M (up from $245M); $110M upfront payment from Nippon Shinyaku collaboration.
- R&D expenses: $53M (down 3% vs. Q1 2024); cash runway extends into H2 2026, potentially longer with non-dilutive financing (e.g., $150M+ PRV sale).

**【Clinical Highlights】**
- RGX-202 Phase I/II: 12 patients across all age groups show >10% microdystrophin expression, functional improvements, and favorable safety profile.
- RGX-121: CAMPSIITE trial met primary endpoint with 80% patients discontinuing ERT; 4-year neurodevelopmental improvements observed.
- RGX-314: Subretinal wet AMD trials demonstrate 4-year durability; suprachoroidal delivery shows shorter steroid regimen (7 weeks) vs. competitors.

**【Q&A Key Takeaways】**
- FDA interactions normal; no signs of accelerated approval pathway changes post-CBER leadership shift.
- RGX-202 manufacturing process (NAVXpress) validated for commercial use; 2,500 doses/year capacity (80%+ full capsid purity).
- AbbVie collaboration progressing on DR Phase III design; first patient dosing in 2025.
- No impact on pricing strategy anticipated from recent branded drug pricing announcements.


REGENXBIO Inc. (RGNX) Q1 2025 Earnings Call Transcript

- **RGX-121 Lead**: First gene therapy BLA for Hunter syndrome; Q4 2025 FDA approval expected with PRV potential.
- **RGX-202 Momentum**: 50% of pivotal enrollment complete; 12-month data shows functional benefits and >80% microdystrophin purity.
- **RGX-314 Expansion**: Wet AMD trials to complete enrollment in 2025; DR pivotal program begins with AbbVie in 2025.
- **Financial Strength**: $245M cash runway to H2 2026; $1.8B AbbVie milestones and PRV monetization support long-term growth.

**【CEO Summary Section】**
- Submitted BLA for RGX-121 (Hunter syndrome) under accelerated approval pathway, targeting Q4 2025 FDA approval.
- RGX-202 (Duchenne muscular dystrophy) pivotal study enrollment is nearly halfway complete, with 30 ambulatory patients (ages 1+). Plans to submit BLA by mid-2026.
- RGX-314 (wet AMD and diabetic retinopathy) advancing with AbbVie: pivotal trials for subretinal wet AMD expected to complete enrollment in 2025, with data in 2026. Suprachoroidal DR pivotal program to begin in 2025.

**【CMO Clinical Update】**
- RGX-202: 12-month data shows functional improvements (e.g., 10m walk test) and robust microdystrophin expression (>80% purity). No serious adverse events reported.
- RGX-314: Suprachoroidal delivery in DR shows favorable safety profile with 7-week steroid regimen. Subretinal wet AMD trials progressing well globally.
- RGX-121: BLA based on CSF heparan sulfate D2S6 reduction to <10% of baseline. 80% of patients discontinued ERT or remained ERT-naïve post-treatment.

**【CFO Financial Overview】**
- Cash balance: $245M as of Q4 2024, supporting operations into H2 2026. Includes $131M from March 2024 equity offering.
- R&D expenses: $209M in 2024, down 9% YoY due to reduced preclinical activity.
- Non-dilutive financing options: 
  - $1.8B AbbVie collaboration milestones (e.g., DR pivotal initiation)
  - Priority Review Voucher monetization
  - Zolgensma royalty reversion ($300M cap)

**【Q&A Highlights】**
- RGX-202 enrollment acceleration: 50% of pivotal study completed, with >180 patients screened. Younger age groups (1-3y) showing higher microdystrophin levels.
- Regulatory confidence: FDA pre-BLA meetings positive; PRV approval probability high for RGX-121.
- Commercial readiness: In-house manufacturing capacity for 2,500 RGX-202 doses/year. Nippon Shinyaku sales team to support RGX-121 launch in US and Asia.
- Wet AMD competition: RGX-314's one-time injection vs. long-acting TKIs; safety profile (7-week steroids vs. 12-16w) a key differentiator.

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